High-level transgene expression in induced pluripotent stem cell–derived megakaryocytes: correction of Glanzmann thrombasthenia
نویسندگان
چکیده
منابع مشابه
Glanzmann thrombasthenia
Glanzmann thrombasthenia (GT) is a rare autosomal recessive bleeding syndrome affecting the megakaryocyte lineage and characterized by lack of platelet aggregation. The molecular basis is linked to quantitative and/or qualitative abnormalities of alphaIIb beta3 integrin. This receptor mediates the binding of adhesive proteins that attach aggregating platelets and ensure thrombus formation at si...
متن کاملGlanzmann thrombasthenia in a neonate.
Glanzmann thrombasthenia is a qualitative platelet function disorder manifested by skin bleeds, epistaxis, gingival bleeding, gastrointestinal hemorrhage, hematuria, hemarthrosis, intracranial hemorrhage and visceral hematomas. We report a six day old newborn presenting with hematuria following suprapubic aspiration, who was diagnosed as Glanzmann thrombasthenia. We believe it to be the younges...
متن کاملGlanzmann Thrombasthenia: A Clinicopathological Profile.
OBJECTIVE To describe the clinical presentation of patients with Glanzmann's thrombasthenia (GT) and evaluate their diagnostic, clinical, and laboratory parameters including platelet aggregometry. STUDY DESIGN Descriptive study. PLACE AND DURATION OF STUDY Department of Hematology and Blood Transfusion, The Children Hospital and Institute of Child Health, Lahore, from January 2006 to Decemb...
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Induced pluripotent stem cells (iPSCs) could be generated by a single gene Oct4 and chemical compounds, in which exogenous expression of Oct4 was indispensable for reprogramming. Recent advances in chemical-mediated cellular reprogramming suggest that small molecules alone (i.e. without Yamanaka factors) can successfully establish iPSCs from mouse somatic cells.
متن کاملMelanocytes Derived from Transgene-Free Human Induced Pluripotent Stem Cells
Odontostomatologiche, Ortodontiche e Chirurgiche, di Oftalmologia, di Patologia della Testa e del Collo, del Cavo Orale e della Comunicazione Audio Verbale e Scienze Ortopediche, Tramautologiche, Riabilitative e Plastico-Ricostruttive, Seconda Università di Napoli, Naples, Italy and Dipartimento di Chimica Farmaceutica e Tossicologica, Facoltà di Farmacia, Università degli Studi di Napoli ‘Fede...
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ژورنال
عنوان ژورنال: Blood
سال: 2014
ISSN: 0006-4971,1528-0020
DOI: 10.1182/blood-2013-10-530725